@article{2764a48d5c6547c9849170445180501d,
title = "Therapeutic Prospects for Friedreich's Ataxia",
abstract = "Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.",
keywords = "frataxin, Friedreich's ataxia, gene therapy, mitochondria, oligonucleotide, oxidative stress",
author = "Siyuan Zhang and Marek Napierala and Napierala, {Jill S.}",
note = "Funding Information: M.N. is in part supported by CRISPR Therapeutics. The sponsor had no role in or influence over the content of this manuscript. Funding Information: We acknowledge the work of our colleagues in contributing to the FRDA treatment pipeline that could not be discussed here owing to space limitations. Work in the laboratory of M.N. and J.S.N. is supported by grants from the National Institute of Neurological Disorders and Stroke , National Institutes of Health ( R01NS081366 and R21 NS101145 to M.N. and R03 NS099953 to J.S.N.), the Friedreich{\textquoteright}s Ataxia Research Alliance , Muscular Dystrophy Association ( MDA418838 ), National Ataxia Foundation , American Academy of Neurology , and by a sponsored research agreement with CRISPR Therapeutics . M.N. is a UAB Pittman Scholar. Funding Information: We acknowledge the work of our colleagues in contributing to the FRDA treatment pipeline that could not be discussed here owing to space limitations. Work in the laboratory of M.N. and J.S.N. is supported by grants from the National Institute of Neurological Disorders and Stroke, National Institutes of Health (R01NS081366 and R21 NS101145 to M.N. and R03 NS099953 to J.S.N.), the Friedreich's Ataxia Research Alliance, Muscular Dystrophy Association (MDA418838), National Ataxia Foundation, American Academy of Neurology, and by a sponsored research agreement with CRISPR Therapeutics. M.N. is a UAB Pittman Scholar. M.N. is in part supported by CRISPR Therapeutics. The sponsor had no role in or influence over the content of this manuscript. Publisher Copyright: {\textcopyright} 2019 Elsevier Ltd",
year = "2019",
month = apr,
doi = "10.1016/j.tips.2019.02.001",
language = "English (US)",
volume = "40",
pages = "229--233",
journal = "Trends in Pharmacological Sciences",
issn = "0165-6147",
publisher = "Elsevier Limited",
number = "4",
}